SpringWorks Therapeutics Inc. said the European Commission granted the orphan-drug designation to its cancer drug nirogacestat.
Nirogacestat is indicated for treating soft tissue sarcoma, a rare type of cancer that begins in the body tissues such as muscles, fats, tendons and nerves. SpringWorks is enrolling patients in a phase 3 study, dubbed DeFi, which will evaluate adult patients with progressing desmoid tumors.
Desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, are rare and often debilitating and disfiguring. They can aggressively attack surrounding healthy tissues and lead to severe pain, internal bleeding, and, in rare cases, death.
An estimated 1,000 to 1,500 new cases are diagnosed annually in the U.S., where there are no therapies approved by the U.S. Food and Drug Administration.
The European Commission grants orphan medicinal status to treatments for life-threatening or very serious conditions that affect no more than five in 10,000 people in the EU. The designation provides companies with certain benefits and incentives in the EU, including a decade of market exclusivity after approval and reduced regulatory fees.
